Navigating the Late Stage Clinical and Regulatory Landscape for Gene Therapies: Assessing the Key Benefits and Challenges of Real World Evidence and Accelerated Approval Pathways
Kaye Spratt, PhD Senior Vice President, Regulatory Abeona Therapeutics Inc
Comparing Manufacturing Methods For Their Suitability For Long Term Commercial Manufacturing
Jude Samulski, University of North Carolina
Gene Therapy Clinical Trails: Can They be Compelling for Patient/Families, Investigators and Health Authorities?
Jonathan Schwartz, MD, Rocket Pharma
We recently conducted an industry-wide survey, gathering together the thoughts and opinions of experts working to address the unmet need in the rare disease space with gene therapies. Here are the results!